Disclosed is a nucleic acid-polypeptide nano-pharmaceutical composition for treating and preventing human papilloma virus infection. A small interfering nucleic acid siRNA molecule used for inhibiting and treating various diseases caused by a HPV infection can block the virus replication life cycle by means of targeted inhibition of the expression of the HP16/18 key gene, reduce a viral infection and finally remove viruses. A pharmaceutical composition based on the siRNA molecule comprises a siRNA molecule and another molecule, including a siRNA molecule for inhibiting PD-1/PD-L1, a small molecule compound against a HPV infection, a therapeutic mRNA/neoantigen vaccine, and the like. The siRNA molecule and other anti-HPV drugs are coupled by means of a specific chemical bond to form a new coupled molecule, and the composition further includes a pharmaceutically acceptable polypeptide polymer nano-introduction carrier. In some embodiments, the carrier is a histidine-lysine polypeptide polymer nanocarrier.
Nucleic acid products are provided that modulate, in particular interfere with or inhibit C3 gene expression. The products can be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from a C3 gene.
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
3.
OLIGONUCLEOTIDE CONSTRUCTS WITH IMPROVED FUNCTION AND THERAPEUTICS
Divalent RNA molecules containing chemically linked siRNA molecules are provided that inhibit the expression of TGFβ1 and cyclooxygenase-2 (Cox-2) genes. The divalent molecules may be used for treating a variety of cancers in a subject, particularly in cells that express Cox-2. Pharmaceutical compositions containing the linked siRNA molecules also are provided.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
A61K 31/7115 - Acides nucléiques ou oligonucléotides ayant des bases modifiées, c.-à-d. autres que l'adénine, la guanine, la cytosine, l'uracile ou la thymine
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 35/00 - Préparations médicinales contenant des substances ou leurs produits de réaction de constitution non déterminée
4.
COMPOSITIONS AND METHODS FOR TREATMENT OF CANCERS USING MODIFIED siRNA-GEM AGENTS
Pharmaceutical compositions are provided comprising gemcitabine (“GEM”)-containing modified RNAi agents such as siRNA molecules, combined with a histidine-lysine copolymer carrier, intended for inhibiting the expression of targeted genes involved in a variety of cancers. Modifications to the siRNA backbone may include, without limitation, 2′-OMe, 2′-Fluoro and phosphorothioate modifications. Methods for treatment of a variety of cancers by administering such pharmaceutical compositions are further provided, including breast, ovarian, and pancreatic cancer in mammals, among others.
Nucleic acid products that modulate, interfere with, or inhibit TMPRSS6 gene expression are provided, together with compositions containing the constructs and methods for their use.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
Nucleic acid products that modulate, interfere with, or inhibit A1 AT gene expression are provided, together with compositions containing the constructs and methods for their use.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61P 1/16 - Médicaments pour le traitement des troubles du tractus alimentaire ou de l'appareil digestif des troubles de la vésicule biliaire ou du foie, p. ex. protecteurs hépatiques, cholagogues, cholélitholytiques
Nucleic acid products that modulate, interfere with, or inhibit ATS gene expression are provided, together with compositions containing the constructs and methods for their use.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61P 7/00 - Médicaments pour le traitement des troubles du sang ou du fluide extracellulaire
Nucleic acid products that modulate, interfere with, or inhibit ANGPTL3 gene expression are provided, together with compositions containing the constructs and methods for their use. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate ANGPTLS-associated disorders such as a disease or disorder being selected from the group consisting of hypertriglyceridemia, obesity, hyperlipidemia, abnormal lipid and/or cholesterol metabolism, atherosclerosis, type II diabetes mellitus, cardiovascular disease, coronary artery disease, dyslipidemia, non-alcoholic steatohepatitis, non-alcoholic fatty liver disease, homozygous and heterozygous familial hypercholesterolemia, and statin resistant hypercholesterolemia.
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61P 3/10 - Médicaments pour le traitement des troubles du métabolisme de l'homéostase du glucose de l'hyperglycémie, p. ex. antidiabétiques
A61P 9/10 - Médicaments pour le traitement des troubles du système cardiovasculaire des maladies ischémiques ou athéroscléreuses, p. ex. médicaments antiangineux, vasodilatateurs coronariens, médicaments pour le traitement de l'infarctus du myocarde, de la rétinopathie, de l'insuffisance cérébro-vasculaire, de l'artériosclérose rénale
Nucleic acid products that modulate, interfere with, or inhibit PCSK9 gene expression are provided, together with compositions containing the constructs and methods for their use.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
A61P 3/10 - Médicaments pour le traitement des troubles du métabolisme de l'homéostase du glucose de l'hyperglycémie, p. ex. antidiabétiques
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
Nucleic acid products that modulate, interfere with, or inhibit HSD17B13 gene expression are provided, together with compositions containing the constructs and methods for their use. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate HSD17B13-associated disorders such as a liver disease or disorder optionally being selected from non-alcoholic fatty liver disease (NAFLD), non-alcoholic seatohepatitis (NASH), alcoholic steatohepatitis (ASH), hepatic fibrosis, steatosis, cirrhosis, in particular alcoholic cirrhosis and/or non-alcoholic cirrhosis, fatty liver disease, chronic liver disease, steatohepatitis, hepatocellular carcinoma, HCV hepatitis, chronic hepatitis, hereditary hemochromatosis, primary sclerosing cholangitis, alcohol-related liver disease (ARLD) and alcoholic fatty liver disease.
C12Q 1/6883 - Produits d’acides nucléiques utilisés dans l’analyse d’acides nucléiques, p. ex. amorces ou sondes pour les maladies provoquées par des altérations du matériel génétique
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
Nucleic acid products are provided that interfere with or inhibit APOC3 and AGT gene expression, together with methods for using the nucleic acids in the treatment of subjects.
Provided are products, and compositions, and their uses. In particular, provided are nucleic acid products that modulate, in particular interfere with or inhibit TTR gene expression.
Double stranded RNAi agents for inhibiting PCSK9 gene expression are provided. Complexes in which the siRNA agents are covalently conjugated to a peptide docking vehicle (PDoV), and further covalently linked to one or more targeting ligands also are provided. Pharmaceutical compositions containing the RNAi agents and complexes are provided, together with methods for their use.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
Oligonucleotides, including siRNA duplex molecules are provided containing one or more antiviral nucleoside analogs. The analogs may be positioned within the oligonucleotide sequence or may be appended to one or more termini of the oligonucleotides. Pharmaceutical compositions containing the oligonucleotides are provided, together with methods of using the compositions for treating viral infections.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
siRNA molecules and pharmaceutical compositions containing siRNA molecules are provided for inhibiting expression of KRAS, RAF1 and mutant KRAS peptides or proteins. Methods of treatment of cancer are provided in which the pharmaceutical compositions are administered to a subject in need thereof. The cancer may be lung, colon, or pancreatic cancers, including non-small cell lung cancer (NSCLC). Combinations of siRNAs, packaged in nanoparticles with co-polymer carriers and delivered simultaneously to target cells, elicit an additive or synergistic effect to inhibit tumorous cell growth.
siRNA molecules and pharmaceutical compositions containing siRNA molecules are provided for inhibiting expression of KRAS, RAF1 and mutant KRAS peptides or proteins. Methods of treatment of cancer are provided in which the pharmaceutical compositions are administered to a subject in need thereof. The cancer may be lung, colon, or pancreatic cancers, including non-small cell lung cancer (NSCLC). Combinations of siRNAs, packaged in nanoparticles with co-polymer carriers and delivered simultaneously to target cells, elicit an additive or synergistic effect to inhibit tumorous cell growth.
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome
C12Q 1/66 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir une luciférase
C12Q 1/6883 - Produits d’acides nucléiques utilisés dans l’analyse d’acides nucléiques, p. ex. amorces ou sondes pour les maladies provoquées par des altérations du matériel génétique
17.
COMBINATION siRNA AND PEPTIDE DOCKING VEHICLE (PDoV) COMPOSITIONS AND METHODS OF THEIR USE FOR IMPROVED REGULATION AND FUNCTIONALITY
siRNA molecules targeting AGT mRNA that reduce or inhibit AGT production are provided. Pharmaceutical compositions containing such siRNA molecules also are provided, together with methods of their use for treating hypertension. Pharmaceutical compositions containing at least one oligonucleotide covalently linked to, and delivered to a target cell by, a peptide docking vehicle (PDoV) are capable of reducing or inhibiting the production of AGT and treating hypertension. The PDoV may contain a targeting ligand such as a GalNAc moiety conjugated to the PDoV which targets the complex to hepatocytes.
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
Methods are provided for prevention and treatment of 2019 coronavirus (2019-nCoV; COVID-19) infections in mammals by prophylactic or therapeutic administration of pharmaceutical compositions known as STP908. STP908 administered intravenously, whether prophylactically or therapeutically, led to survival of 50 percent of the treatment group infected with 2019-nCoV. STP908 compositions and methods of making them have been previously disclosed, and comprise potent siRNA therapeutics formulated in a histidine-lysine polymeric carrier; the siRNA molecules in STP908 target and reduce or inhibit the expression of two genes of the 2019-nCoV genome: ORF1AB and N-protein, preventing or ameliorating COVID-19 symptoms.
Nucleic acid products, compositions and related methods of use are provided that modulate, in particular, interfere with or inhibit CFB and CS gene expression in mammals and are useful to treat, prevent, or ameliorate CFB- and CS-associated disorders. Nucleic acid products and compositions are provided, together with methods for their use, to modulate, in particular, interfere with or inhibit CFB and CS gene expression.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/711 - Acides désoxyribonucléiques naturels, c.-à-d. contenant uniquement des 2'-désoxyriboses liés à l'adénine, la guanine, la cytosine ou la thymine et ayant des liaisons 3'-5' phosphodiester
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
Nucleic acid products are provided that modulate, in particular interfere with or inhibit AGT gene expression. The products can be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from an AGT gene, wherein said first nucleobase sequence is selected from SEQ ID NOs 1 to 100.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
siRNA molecules targeting AGT mRNA that reduce or inhibit AGT production are provided. Pharmaceutical compositions containing such siRNA molecules also are provided, together with methods of their use for treating hypertension. Pharmaceutical compositions containing at least one oligonucleotide covalently linked to, and delivered to a target cell by, a peptide docking vehicle (PDoV) are capable of reducing or inhibiting the production of AGT and treating hypertension. The PDoV may contain a targeting ligand such as a GalNAc moiety conjugated to the PDoV which targets the complex to hepatocytes.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61K 31/7115 - Acides nucléiques ou oligonucléotides ayant des bases modifiées, c.-à-d. autres que l'adénine, la guanine, la cytosine, l'uracile ou la thymine
The present disclosure relates to products, and compositions, and their uses. In particular, the present disclosure relates to nucleic acid products that modulate, in particular interfere with or inhibit C5 gene expression. The products can be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from a C5 gene.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
Nucleic acid products are provided that modulate, in particular interfere with or inhibit C3 gene expression. The products can be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from a C3 gene.
Nucleic acid products are provided that modulate, in particular interfere with or inhibit, TMPRSS6 and APOC3 gene expression. Embodiments of the present disclosure can provide methods, compounds, and compositions for reducing expression of TMPRSS6 and APOC3 mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate TMPRSS6- and APOC3-associated disorders such as iron overload or hemochromatosis, and dyslipidaemia.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
A61K 31/7115 - Acides nucléiques ou oligonucléotides ayant des bases modifiées, c.-à-d. autres que l'adénine, la guanine, la cytosine, l'uracile ou la thymine
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
Pharmaceutical constructs containing a Peptide Docking Vehicle (PDoV) covalently linked to: (a) a targeting moiety; and (b) a first therapeutic nucleic acid, are provided, where the therapeutic nucleic acid inhibits replication of Hepatitis B virus (HBV). Methods of using the constructs for treating HBV in subjects also are provided.
Methods are provided for inducing the remodeling of adipose tissue in patients to improve health as well as the aesthetic appearance of adipose tissue. RNAi techniques are used with transforming growth factor beta 1 (TGF-β1) and cyclooxygenase 2 (Cox2), both of which have been implicated in disease progression and negative remodeling effects on skin tissues. Administration of two siRNAs targeting TGF-β1 and Cox2 in a single nanoparticle formulation permits entry of the siRNAs into the same cells at the same time and the silencing of target genes in these cells results in favorable remodeling of adipose tissue.
siRNA sequences for inhibiting TGFβ and Cox-2 gene expression are provided. Methods for treatment of skin cancers, in which pharmaceutical compositions or containing these siRNA agents and complexes, are further provided, in particular, for treating squamous cell carcinoma (isSCC) and/or basal cell carcinoma (BCC). TGFβ and Cox-2 have each been implicated in driving cancer progression. TGFβ is upregulated in a number of tumor types and plays a role in stimulating cancer-associated fibroblast development. Cox-2 upregulation plays a negative role in inducing inflammation and converting active T-cells to inactive T-reg cells. Co-delivery of the two siRNAs into the same cell at the same time silences of both targets at the same time results in antitumoral activity.
C07K 16/28 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
28.
COMPOSITION AND USE OF siRNAs AGAINST VEGFR2 AND TGF-BETA1 IN COMBINATION THERAPY FOR CANCER
The techniques of the present disclosure provide a method of inhibiting tumor growth in a tissue of a mammal. The method includes administering to the mammal a therapeutically effective amount of a composition comprising an siRNA molecule that binds to an mRNA that codes for TGFβ1 protein, an siRNA molecule that binds to an mRNA that codes for VEGFR2 protein, and a pharmaceutically acceptable carrier comprising a pharmaceutically acceptable polypeptide polymer. The techniques of the present disclosure also provide for additional methods for using this composition.
Pharmaceutical compositions are provided comprising gemcitabine ("GEM")-containing modified RNAi agents such as siRNA molecules, combined with a histidine-lysine copolymer carrier, intended for inhibiting the expression of targeted genes involved in a variety of cancers. Modifications to the siRNA backbone may include, without limitation, 2'-OMe, 2'-Fluoro and phosphorothioate modifications. Methods for treatment of a variety of cancers by administering such pharmaceutical compositions are further provided, including breast, ovarian, and pancreatic cancer in mammals, among others.
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
G01N 33/574 - Tests immunologiquesTests faisant intervenir la formation de liaisons biospécifiquesMatériaux à cet effet pour le cancer
G01N 33/00 - Recherche ou analyse des matériaux par des méthodes spécifiques non couvertes par les groupes
Nucleic acid products that modulate, interfere with, or inhibit PCSK9 and APOC3 gene expression are provided, together with compositions containing the constructs and methods for their use.
Methods are provided for prevention and treatment of 2019 coronavirus (2019-nCoV; COVID-19) infections in mammals by prophylactic or therapeutic administration of pharmaceutical compositions known as STP707, which compositions have been previously disclosed. These compositions comprise potent siRNA therapeutics formulated in a histidine-lysine polymeric carrier; the siRNA molecules target and reduce or inhibit TGFβ1 and Cox2 gene expression, preventing or ameliorating COVID-19 symptoms.
Methods of making and using nanoparticle pharmaceutical compositions comprising histidine-lysine copolymers are provided. The solutions spontaneously form nanoparticles when mixed with nucleic acids such as siRNA. Methods are provided where the pH of the nucleic acid solution is controlled prior to mixing leading to a reduction in nanoparticle diameter to a desirable range, typically 100-150 nm, and Polydispersity Index (PDI), both of which improve transport into target cells to improve the efficacy of gene silencing.
Methods are provided for inducing the remodeling of adipose tissue in patients to improve health as well as the aesthetic appearance of adipose tissue. RNAi techniques are used with transforming growth factor beta 1 (TGF-β1) and cyclooxygenase 2 (Cox2), both of which have been implicated in disease progression and negative remodeling effects on skin tissues. Administration of two siRNAs targeting TGF-β1 and Cox2 in a single nanoparticle formulation permits entry of the siRNAs into the same cells at the same time and the silencing of target genes in these cells results in favorable remodeling of adipose tissue.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
B82Y 5/00 - Nanobiotechnologie ou nanomédecine, p. ex. génie protéique ou administration de médicaments
B82Y 30/00 - Nanotechnologie pour matériaux ou science des surfaces, p. ex. nanocomposites
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
34.
TREATMENT OF 2019-NCOV USING SIRNAS AGAINST TGFB1 AND COX2
Methods are provided for prevention and treatment of 2019 coronavirus (2019-nCoV; COVID-19) infections in mammals by prophylactic or therapeutic administration of pharmaceutical compositions known as STP707, which compositions have been previously disclosed. These compositions comprise potent siRNA therapeutics formulated in a histidine-lysine polymeric carrier; the siRNA molecules target and reduce or inhibit TGF01 and Cox2 gene expression, preventing or ameliorating COVID-19 symptoms.
Methods are provided for prevention and treatment of 2019 coronavirus (2019-nCoV; COVID-19) infections in mammals by prophylactic or therapeutic administration of pharmaceutical compositions known as STP908. STP908 administered intravenously, whether prophylactically or therapeutically, led to survival of 50 percent of the treatment group infected with 2019-nCoV. STP908 compositions and methods of making them have been previously disclosed, and comprise potent siRNA therapeutics formulated in a histidine- lysine polymeric carrier; the siRNA molecules in STP908 target and reduce or inhibit the expression of two genes of the 2019-nCoV genome: ORF1AB and N-protein, preventing or ameliorating COVID-19 symptoms.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61P 11/00 - Médicaments pour le traitement des troubles du système respiratoire
A61P 31/14 - Antiviraux pour le traitement des virus ARN
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
Nucleic acid products that modulate, in particular interfere with or inhibit CFB gene expression, are provided. The products may be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion o 5 f RNA transcribed from a CFB gene, where the first nucleobase sequence is selected from SEQ ID NOs 1 to 391, 751 and 752, or a fragment thereof.
Nucleic acid products that modulate, interfere with, or inhibit PCSK9 and APOC3 gene expression are provided, together with compositions containing the constructs and methods for their use.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7115 - Acides nucléiques ou oligonucléotides ayant des bases modifiées, c.-à-d. autres que l'adénine, la guanine, la cytosine, l'uracile ou la thymine
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
C07H 21/00 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques
38.
METHODS FOR PREPARING NANOPARTICLE COMPOSITIONS CONTAINING HISTIDINE-LYSINE COPOLYMERS
Improved pharmaceutical nanoparticle compositions and improved methods for preparing the compositions comprising histidine-lysine copolymers and an acetate salt or phosphate anion are provided. The addition of acetate or phosphate anion to the histidine-lysine copolymer prior to mixing with a nucleic acid alters nanoparticle size and polydispersity index of the compositions and provides a more uniform particle size distribution.
Improved pharmaceutical nanoparticle compositions and improved methods for preparing the compositions comprising histidine-lysine copolymers and an acetate salt or phosphate anion are provided. The addition of acetate or phosphate anion to the histidine-lysine copolymer prior to mixing with a nucleic acid alters nanoparticle size and polydispersity index of the compositions and provides a more uniform particle size distribution.
A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
40.
NANOPARTICLE FORMULATIONS FORMED FROM HISTIDINE-LYSINE COPOLYMERS
Methods are provided for improving the manufacture and use of pharmaceutical compositions comprising histidine-lysine copolymers and nucleic acids, which spontaneously form nanoparticles when mixed. The flow rate of mixing and the ratio of copolymer to siRNA strongly affect nanoparticle properties, including size and homogeneity of particles, resulting in greater efficacy in delivery to target cells. Further, an acidic pH of the siRNA solution, as well as the addition of acetate or phosphate salt to the histidine-lysine copolymer prior to mixing with the siRNA also contribute to lower nanoparticle diameters and more uniform particles (lower PDI).
A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
41.
NANOPARTICLE PHARMACEUTICAL COMPOSITIONS WITH REDUCED NANOPARTICLE SIZE AND IMPROVED POLYDISPERSITY INDEX
Methods of making and using nanoparticle pharmaceutical compositions comprising histidine-lysine copolymers are provided. The solutions spontaneously form nanoparticles when mixed with nucleic acids such as siRNA. Methods are provided where the pH of the nucleic acid solution is controlled prior to mixing leading to a reduction in nanoparticle diameter to a desirable range, typically 100-150 nm, and Poly dispersity Index (PDI), both of which improve transport into target cells to improve the efficacy of gene silencing.
A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
A61K 47/34 - Composés macromoléculaires obtenus par des réactions autres que celles faisant intervenir uniquement des liaisons non saturées carbone-carbone, p. ex. polyesters, acides polyaminés, polysiloxanes, polyphosphazines, copolymères de polyalkylène glycol ou de poloxamères
Methods are provided for improving the manufacture and use of pharmaceutical compositions comprising histidine-lysine copolymers and nucleic acids, which spontaneously form nanoparticles when mixed. The flow rate of mixing and the ratio of copolymer to siRNA strongly affect nanoparticle properties, including size and homogeneity of particles, resulting in greater efficacy in delivery to target cells. Further, an acidic pH of the siRNA solution, as well as the addition of acetate or phosphate salt to the histidine-lysine copolymer prior to mixing with the siRNA also contribute to lower nanoparticle diameters and more uniform particles (lower PDI).
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
B82Y 5/00 - Nanobiotechnologie ou nanomédecine, p. ex. génie protéique ou administration de médicaments
B82Y 40/00 - Fabrication ou traitement des nanostructures
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
Nucleic acid products and compositions and their uses are provided. In particular, nucleic acid products are provided that modulate, interfere with, or inhibit APOC3 gene expression. The products can be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from a APOC3 gene, wherein said first nucleobase sequence is selected from the following sequences, or a portion thereof: SEQ ID NOs 1 to 39
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 31/232 - Esters, p. ex. nitroglycérine, sélénocyanates d'acides carboxyliques d'acides acycliques, p. ex. pravastatine d'acides ayant un groupe carboxyle lié à une chaîne d'au moins sept atomes de carbone ayant au moins trois doubles liaisons, p. ex. étrétinate
A61K 31/397 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines ayant des cycles à quatre chaînons, p. ex. azétidine
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
Nucleic acid products are provided that modulate, interfere with or inhibit PCSK9 gene expression. The products include compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from a PCSK9 gene, wherein the first nucleobase sequence is selected from the following sequences, or a portion thereof: SEQ ID NOs 1 to 250 or 501 to 543.
Nucleic acid products that modulate, in particular interfere with or inhibit CFB gene expression, are provided. The products may be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion o 5 f RNA transcribed from a CFB gene, where the first nucleobase sequence is selected from SEQ ID NOs 1 to 391, 751 and 752, or a fragment thereof.
The techniques of the present disclosure provide a method of inhibiting tumor growth in a tissue of a mammal. The method includes administering to the mammal a therapeutically effective amount of a composition comprising an siRNA molecule that binds to an mRNA that codes for TGF01 protein, an siRNA molecule that binds to an mRNA that codes for VEGFR2 protein, and a pharmaceutically acceptable carrier comprising a pharmaceutically acceptable polypeptide polymer. The techniques of the present disclosure also provide for additional methods for using this composition.
A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
B82Y 5/00 - Nanobiotechnologie ou nanomédecine, p. ex. génie protéique ou administration de médicaments
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
47.
SIRNA-COPOLYMER COMPOSITIONS AND METHODS OF USE FOR TREATMENT OF LIVER CANCER
Compositions and methods for treating hepatocellular carcinoma (HCC) using siRNA molecules are provided. The compositions advantageously are administered in nanoparticle form, where the nanoparticles also contain a histidine-lysine copolymer ("HKP"). In specific embodiments, the composition contains an siRNA molecule that targets TGF-βi, an siRNA molecule that targets Cox-2, and an HKP copolymer.
Nucleic acid products and compositions and their uses are provided. In particular, nucleic acid products are provided that modulate, interfere with, or inhibit APOC3 gene expression. The products can be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from a APOC3 gene, wherein said first nucleobase sequence is selected from the following sequences, or a portion thereof: SEQ ID NOs 1 to 39.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
Compositions and methods are provided for the silencing of the NSD3 gene. Specifically, siRNA compositions are provided that contain siRNA molecules that target the wild-type NSD3 gene or the NSD3T1232A mutant. Methods for using these compositions for treating cancer also are provided.
Nucleic acid products are provided that modulate, interfere with or inhibit PCSK9 gene expression. The products include compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from a PCSK9 gene, wherein the first nucleobase sequence is selected from the following sequences, or a portion thereof: SEQ ID NOs 1 to 250 or 501 to 543.
siRNA compositions are provided that contain gemcitabine (GEM) in place of cytosine moieties within the siRNA sequence. Pharmaceuticals compositions containing these siRNA molecules, and methods of using the compositions for treating diseases such as cancer are provided.
A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
A61K 47/55 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique l’agent de modification étant aussi un agent pharmacologiquement ou thérapeutiquement actif, c.-à-d. le conjugué entier étant un co-médicament, p. ex. un dimère, un oligomère ou un polymère de composés pharmacologiquement ou thérapeutiquement actifs
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
Disclosed is a nucleic acid-polypeptide nano-pharmaceutical composition for treating and preventing human papilloma virus infection. A small interfering nucleic acid siRNA molecule used for inhibiting and treating various diseases caused by a HPV infection can block the virus replication life cycle by means of targeted inhibition of the expression of the HP16/18 key gene, reduce a viral infection and finally remove viruses. A pharmaceutical composition based on the siRNA molecule comprises a siRNA molecule and another molecule, specifically a siRNA molecule for inhibiting PD-1/PD-L1, a small molecule compound against a HPV infection, a therapeutic mRNA/neoantigen vaccine, etc. The siRNA molecule and other anti-HPV drugs are coupled by means of a specific chemical bond to form a new coupled molecule, and the composition further comprises a pharmaceutically acceptable polypeptide polymer nano-introduction carrier, and the carrier is preferably a histidine-lysine polypeptide polymer nanocarrier.
A61K 47/55 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique l’agent de modification étant aussi un agent pharmacologiquement ou thérapeutiquement actif, c.-à-d. le conjugué entier étant un co-médicament, p. ex. un dimère, un oligomère ou un polymère de composés pharmacologiquement ou thérapeutiquement actifs
A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
A61K 47/42 - ProtéinesPolypeptidesLeurs produits de dégradationLeurs dérivés p. ex. albumine, gélatine ou zéine
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
Products, compositions, and their uses are provided. In particular, nucleic acid products that modulate, in particular interfere with or inhibit, Factor XI (FXI) gene expression are provided. The products can be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from an FXI gene, wherein said first nucleobase sequence is selected from the following sequences, or a portion thereof: SEQ ID NOs 1 to 250.
Products, compositions, and their uses are provided. In particular, nucleic acid products that modulate, in particular interfere with or inhibit, Factor XI (FXI) gene expression are provided. The products can be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from an FXI gene, wherein said first nucleobase sequence is selected from the following sequences, or a portion thereof: SEQ ID NOs 1 to 250.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61P 1/16 - Médicaments pour le traitement des troubles du tractus alimentaire ou de l'appareil digestif des troubles de la vésicule biliaire ou du foie, p. ex. protecteurs hépatiques, cholagogues, cholélitholytiques
A61P 29/00 - Agents analgésiques, antipyrétiques ou anti-inflammatoires non centraux, p. ex. agents antirhumatismauxMédicaments anti-inflammatoires non stéroïdiens [AINS]
A61P 43/00 - Médicaments pour des utilisations spécifiques, non prévus dans les groupes
55.
COMBINATIONS OF SIRNAS WITH SIRNAS AGAINST SULF2 OR GPC3 FOR USE IN TREATING CANCER
Compositions and methods are provided for silencing the Sulf2 and/or GPC3 genes in vivo. Potent siRNA sequences are provided that silence regions of the Sulf2 and GPC3 genes that are identical in human, mice and non-human primates. Combinations of siRNAs also are provided that result in additivity or synergy with silencing of Sulf2 and/or GPC3. Silencing SULF2+TGFβ1 showed a dramatic effect against cancer growth in vitro and in vivo.
A novel nucleic acid delivery system is provided containing a linear histidine-lysine rich cysteine-containing peptide bearing a targeting function, and a four branched histidine-lysine rich polypeptide. The delivery system includes a nucleic acid, such as an siRNA. The components form a nanoparticle complex through multiple non-covalent interactions between the phosphates of siRNA and histidine/lysine of the polypeptide, with reduced toxicity. The stable complex selectively delivers the genetic material to cells. The targeting function enhances the efficiency of the nucleic acid delivery and transfection.
A novel nucleic acid delivery system is provided containing a linear histidine-lysine rich cysteine-containing peptide bearing a targeting function, and a four branched histidine-lysine rich polypeptide. The delivery system includes a nucleic acid, such as an siRNA. The components form a nanoparticle complex through multiple non-covalent interactions between the phosphates of siRNA and histidine/lysine of the polypeptide, with reduced toxicity. The stable complex selectively delivers the genetic material to cells. The targeting function enhances the efficiency of the nucleic acid delivery and transfection.
Carrier molecules also are provided that have the ability to deliver a therapeutic molecule to a specific cell within a tissue in the body. The carrier molecule is modified with a targeting ligand capable of binding to specific receptors present or upregulated on the cell to be targeted. The therapeutic molecule is an siRNA, miRNA, or other oligonucleotide. The targeting moiety is a small molecule, peptide, or protein that shows an affinity for a receptor present on the cell to be targeted.
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
A61K 47/55 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique l’agent de modification étant aussi un agent pharmacologiquement ou thérapeutiquement actif, c.-à-d. le conjugué entier étant un co-médicament, p. ex. un dimère, un oligomère ou un polymère de composés pharmacologiquement ou thérapeutiquement actifs
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/115 - Aptamères, c.-à-d. acides nucléiques liant spécifiquement une molécule cible avec une haute affinité sans s'y hybrider
57.
COMBINATIONS OF SIRNAS WITH SIRNAS AGAINST SULF2 OR GPC3 FOR USE IN TREATING CANCER
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 47/62 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant une protéine, un peptide ou un acide polyaminé
Compositions and methods are provided for the silencing of the BCLxL and MCL1 genes. Specifically, siRNA compositions are provided that contain siRNA molecules that target the BCLxL and MCL1 genes. Methods for using these compositions for treating cancer also are provided.
A61K 31/70 - Hydrates de carboneSucresLeurs dérivés
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61K 35/12 - Substances provenant de mammifèresCompositions comprenant des tissus ou des cellules non spécifiésCompositions comprenant des cellules souches non embryonnairesCellules génétiquement modifiées
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
59.
METHODS OF CANCER TREATMENT BY DELIVERY OF SIRNAS AGAINST NSD3
Compositions and methods are provided for the silencing of the NSD3 gene. Specifically, nanoparticle siRNA compositions are provided that contain siRNA molecules that target the wild-type NSD3 gene or the NSD3n232A mutant. Methods for using these compositions for treating cancer by administering an effective amount of a nanoparticle composition also are provided.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12Q 1/68 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir des acides nucléiques
G16B 25/10 - Profilage de l’expression de gènes ou de protéinesEstimation ou normalisation de ratio d’expression
60.
METHODS OF CANCER TREATMENT BY DELIVERY OF siRNAs AGAINST BCLXL AND MCL1 USING A POLYPEPTIDE NANOPARTICLE
Compositions and methods are provided for the silencing of the BCLxL and MCL1 genes. Specifically, siRNA compositions are provided that contain siRNA molecules that target the BCLxL and MCL1 genes. Methods for using these compositions for treating cancer also are provided.
A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
Compositions containing an anti-TGF-β siRNA molecule and an anti-PDL1 siRNA molecule are provided. Methods of using these compositions to treat cancer also are provided.
Pharmaceutical constructs containing a Peptide Docking Vehicle (PDoV) covalently linked to: (a) a targeting moiety; and (b) a first therapeutic nucleic acid, are provided, where the therapeutic nucleic acid inhibits replication of Hepatitis B virus (HBV). Methods of using the constructs for treating HBV in subjects also are provided.
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 31/20 - Antiviraux pour le traitement des virus ADN
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
63.
COMPOSITIONS AND METHODS FOR TREATMENT OF SKIN CANCERS
siRNA sequences for inhibiting ΤGFβ and Cox-2 gene expression are provided. Methods for treatment of skin cancers, in which pharmaceutical compositions or containing these siRNA agents and complexes, are further provided, in particular, for treating squamous cell carcinoma (isSCC) and/or basal cell carcinoma (BCC). ΤGFβ and Cox-2 have each been implicated in driving cancer progression. ΤGFβ is upregulated in a number of tumor types and plays a role in stimulating cancer-associated fibroblast development. Cox-2 upregulation plays a negative role in inducing inflammation and converting active T-cells to inactive T-reg cells. Co-delivery of the two siRNAs into the same cell at the same time silences of both targets at the same time results in anti tumoral activity.
Double stranded RNAi agents for inhibiting PCSK9 gene expression are provided. Complexes in which the siRNA agents are covalently conjugated to a peptide docking vehicle (PDoV), and further covalently linked to one or more targeting ligands also are provided. Pharmaceutical compositions containing the RNAi agents and complexes are provided, togetherwith methods for their use.
Oligonucleotides, including siRNA duplex molecules are provided containing one or more antiviral nucleoside analogs. The analogs may be positioned within the oligonucleotide sequence or may be appended to one or more termini of the oligonucleotides. Pharmaceutical compositions containing the oligonucleotides are provided, together with methods of using the compositions for treating viral infections.
A61K 31/00 - Préparations médicinales contenant des ingrédients actifs organiques
A61K 31/70 - Hydrates de carboneSucresLeurs dérivés
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61K 38/00 - Préparations médicinales contenant des peptides
A61K 45/00 - Préparations médicinales contenant des ingrédients actifs non prévus dans les groupes
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
66.
Multi-Targeting Nucleic Acid Constructs Composed Of Multiple Oligonucleotides That Modulate Gene Expression Through Complimentary Interactions With Targets
The present invention provides a multi-targeting nucleic acid construct comprising at least: (a) a first nucleic acid portion that is at least partially complementary to at least a first portion of RNA transcribed from a target gene; (b) a second nucleic acid portion that is at least partially complementary to at least a second portion of RNA transcribed from a target gene, which target gene may be the same or different to the target gene defined in (a); (c) a third nucleic acid portion that is at least partially complementary to the first nucleic acid portion of (a), so as to form a first nucleic acid duplex region therewith; (d) a fourth nucleic acid portion that is at least partially complementary to said second nucleic acid portion of (b), so as to form a second nucleic acid duplex region therewith. The construct is designed so that subsequent to in vivo administration the construct disassembles to yield at least first and second discrete nucleic acid targeting molecules that respectively target RNA transcribed from the target genes of (a) and (b). Typically, the first nucleic acid targeting molecule is capable of modulating expression of the target gene of (a), and comprises, or is derived from, at least the first nucleic acid portion of (a). Typically, the second nucleic acid targeting molecule is capable of modulating expression of said target gene of (b), and comprises, or is derived from, the second nucleic acid portion of (b).
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
67.
MINIATURIZED HAIRPIN RNAi TRIGGERS (mxRNA) AND METHODS OF USES THEREOF
The present invention relates to novel RNAi triggers that can be chemically synthesized and used to modulate gene expression inside animal cells to study various genes function in laboratories or as an active ingredient for agricultural, veterinary, cosmetic and/or therapeutic applications
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
68.
SILENCING TGF-BETA 1 and COX2 USING siRNAs DELIVERED in a POLYPEPTIDE NANOPARTICLE ALONE and in COMBINATION with IMMUNE CHECKPOINT INHIBITORS to TREAT CANCER
The present invention provides certain pharmaceutical molecules and compositions and methods of using them to treat cancer. The molecules are small interfering RNA (siRNA) molecules that inhibit TGF-beta 1 and Cox2 in humans and other mammals, which are used alone or in combination with immune checkpoint inhibitors, to treat cancer.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
C07K 16/28 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
Composition and methods of RNAi prophylactics and therapeutics for treatment of severe acute respiratory infection caused by 2019 novel coronavirus (2019-nCoV)
Compositions and methods for development of potent siRNA therapeutics for prevention and treatment of Corona Virus (2019-nCoV; COVID-19) infections are provided. The compositions include a pharmaceutical composition comprising siRNA cocktails that target critical viral genes and pharmaceutically acceptable polymeric nanoparticle carriers and liposomal nanoparticle carriers. Administration methods for prevention and treatment are provided, including airway instillation, subcutaneous injections and nebulizer aerosolization.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 47/28 - Stéroïdes, p. ex. cholestérol, acides biliaires ou acide glycyrrhétinique
A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
A61P 31/14 - Antiviraux pour le traitement des virus ARN
70.
COMPOSITION AND METHODS OF RNAi PROPHYLACTICS AND THERAPEUTICS FOR TREATMENT OF SEVERE ACUTE RESPIRATORY INFECTION CAUSED BY 2019 NOVEL CORONAVIRUS (2019-nCoV)
Compositions and methods for development of potent siRNA therapeutics for prevention and treatment of Corona Virus (2019-nCoV; COVID-19) infections are provided. The compositions include a pharmaceutical composition comprising siRNA cocktails that target critical viral genes and pharmaceutically acceptable polymeric nanoparticle carriers and liposomal nanoparticle carriers. Administration methods for prevention and treatment are provided, including airway instillation, subcutaneous injections and nebulizer aerosolization.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Drug delivery agents in the form of a coating for nanoparticle polymers that facilitate the delivery of a wide range of pharmaceuticals; Pharmaceutical products for the treatment of viral and infectious diseases, for the treatment of cancer
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Drug delivery agents in the form of a coating for nanoparticle polymers that facilitate the delivery of a wide range of pharmaceuticals; Pharmaceutical products for the treatment of viral and infectious diseases, for the treatment of cancer
73.
Peptide docking vehicle for targeted nucleic acid delivery
Peptide docking vehicle compositions containing a therapeutic compound, such as an siRNA molecule, and a targeting ligand are provided, together with methods for their preparation and use. The compositions and methods allow targeted cell/tissue delivery of the therapeutic compound to a subject by linking a targeting ligand to the compound to provide enhanced therapeutic benefit. The subject may be an animal or a human.
C07K 7/08 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 12 à 20 amino-acides
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
C07K 7/06 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 5 à 11 amino-acides
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/117 - Acides nucléiques présentant des propriétés immunomodulatrices, p. ex. contenant des motifs CpG
74.
PEPTIDE DOCKING VEHICLE FOR TARGETED NUCLEIC ACID DELIVERY
Peptide docking vehicle compositions containing a therapeutic compound, such as an siRNA molecule, and a targeting ligand are provided, together with methods for their preparation and use. The compositions and methods allow targeted cell/tissue delivery of the therapeutic compound to a subject by linking a targeting ligand to the compound to provide enhanced therapeutic benefit. The subject may be an animal or a human.
The present invention provides certain peptides and polypeptides useful in the preparation of nanoparticles for delivering nucleic acids and pharmaceutical drugs to mammalian cells and to humans and other mammals. It further provides methods for making the peptides, polypeptides, and nanoparticles and methods for using the nanoparticles.
A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
C07K 4/00 - Peptides ayant jusqu'à 20 amino-acides dans une séquence indéterminée ou partiellement déterminéeLeurs dérivés
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
76.
TUMOR-TARGETING POLYPEPTIDE NANOPARTICLE DELIVERY SYSTEM FOR NUCLEIC ACID THERAPEUTICS
A novel nucleic acid delivery system is provided containing a linear histidine-lysine rich cysteine-containing peptide bearing a targeting function, and a four branched histidinelysine rich polypeptide. The delivery system includes nucleic acid such as an siRNA. The components form a stable nanoparticle complex through non-covalent interactions between the phosphates of siRNA and histidine/lysine of the polypeptide, with reduced toxicity, and selectively delivers the genetic material to cells. The targeting function enhances the efficiency of the nucleic acid delivery and transfection. Carrier molecules are provided that able to deliver a therapeutic molecule to a specific cell. The carrier molecule is modified with a targeting ligand capable of binding to specific receptors on the cell to be targeted. The therapeutic molecule is an siRNA, miRNA, or other oligonucleotide. The targeting moiety is a small molecule, peptide, or protein that shows an affinity for a receptor present on the cell to be targeted.
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
A61K 47/65 - Séquences de liaison, liants ou bras-espaceurs peptidiques, p. ex. séquences de liaison peptidiques vulnérable aux protéases
C07K 7/08 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 12 à 20 amino-acides
siRNA compositions are provided that contain gemcitabine (GEM) in place of cytosine moieties within the siRNA sequence. Pharmaceuticals compositions containing these siRNA molecules, and methods of using the compositions for treating diseases such as cancer are provided.
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
Compositions containing an anti-TGF-β siRNA molecule and an anti-PDL1 siRNA molecule are provided. Methods of using these compositions to treat cancer also are provided. The anti-TGF-β siRNA molecule may contain an anti-TGF-βΙ siRNA molecule. One or both molecules may comprise an oligonucleotide with a length of 19 base pairs to 25 base pairs, and one or both may be chemically modified to increase their stability.
Sirnaomics Medicine Technology (Suzhou) Co, Ltd. (Chine)
Inventeur(s)
Xu, John
Lu, Patrick Y.
Zhou, Jia
Li, Qingfeng
Simonenko, Vera
Abrégé
The current invention provides a method of activating fibroblast and myofibroblast apoptosis in a tissue of a mammal, comprising administering to the tissue a therapeutically effective amount of a composition comprising an siRNA molecule that binds to an mRNA that codes for TGFB1 protein in a mammalian cell, an siRNA molecule that binds to an mRNA that codes for COX-2 protein in a mammalian cell, and a pharmaceutically acceptable carrier comprising a pharmaceutically acceptable histidine-lysine polymer. The invention also provides additional methods for using this composition.
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 47/34 - Composés macromoléculaires obtenus par des réactions autres que celles faisant intervenir uniquement des liaisons non saturées carbone-carbone, p. ex. polyesters, acides polyaminés, polysiloxanes, polyphosphazines, copolymères de polyalkylène glycol ou de poloxamères
The invention relates to the targeted delivery of therapeutic molecules to organs, tissues, and cells of humans and other mammals. The invention is directed to a chemical construct for delivering such therapeutic molecules and to methods of making and using the constructs.
The present invention provides certain pharmaceutical molecules and compositions and methods of using them to treat cancer. The molecules are small interfering RNA (siRNA) molecules that inhibit TGF-beta 1 and Cox2 in humans and other mammals, which are used alone or in combination with immune checkpoint inhibitors, to treat cancer.
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
82.
COMPOSITION AND METHODS OF CONTROLLABLE CO-COUPLING POLYPEPTIDE NANOPARTICLE DELIVERY SYSTEM FOR NUCLEIC ACID THERAPEUTICS
The present invention provides certain peptides and polypeptides useful in the preparation of nanoparticles for delivering nucleic acids and pharmaceutical drugs to mammalian cells and to humans and other mammals. It further provides methods for making the peptides, polypeptides, and nanoparticles and methods for using the nanoparticles.
A01N 43/04 - Biocides, produits repoussant ou attirant les animaux nuisibles, ou régulateurs de croissance des végétaux, contenant des composés hétérocycliques comportant des cycles avec un ou plusieurs atomes d'oxygène ou de soufre comme uniques hétéro-atomes du cycle avec un hétéro-atome
A61K 31/70 - Hydrates de carboneSucresLeurs dérivés
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61K 35/12 - Substances provenant de mammifèresCompositions comprenant des tissus ou des cellules non spécifiésCompositions comprenant des cellules souches non embryonnairesCellules génétiquement modifiées
83.
Sirna/nanoparticle formulations for treatment of middle-east respiratory syndrome coronaviral infection
The present invention relates to compositions and methods for siRNA therapeutics for prevention and treatment of Middle East Respiratory Syndrome Corona Virus (MERS-CoV) infections. The compositions include a pharmaceutical composition comprising siRNA cocktails that target viral genes and pharmaceutically acceptable polymeric nanoparticle carriers and liposomal nanoparticle carriers.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique
A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
A61P 31/14 - Antiviraux pour le traitement des virus ARN
A61K 47/28 - Stéroïdes, p. ex. cholestérol, acides biliaires ou acide glycyrrhétinique
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques
B82Y 5/00 - Nanobiotechnologie ou nanomédecine, p. ex. génie protéique ou administration de médicaments
The present invention provides pharmaceutical compositions comprising the chemotherapy drug gemcitabine (GEM) and certain derivatives, a taurocholic acid (TCA) formulation, and a Histidine-Lysine Polymer (HKP) conjugate, for enhancement of RNAi cancer therapeutics.
A61K 31/7068 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p. ex. nucléosides, nucléotides contenant des cycles à six chaînons avec l'azote comme hétéro-atome d'un cycle contenant des pyrimidines condensées ou non-condensées ayant des groupes oxo liés directement au cycle pyrimidine, p. ex. cytidine, acide cytidylique
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
85.
PHARMACEUTICAL COMPOSITIONS AND METHODS OF USE FOR ACTIVATION OF HUMAN FIBROBLAST AND MYOFIBROBLAST APOPTOSIS
The current invention provides a method of activating fibroblast and myofibroblast apoptosis in a tissue of a mammal, comprising administering to the tissue a therapeutically effective amount of a composition comprising an siRNA molecule that binds to an mRNA that codes for TGFBl protein in a mammalian cell, an siRNA molecule that binds to an mRNA that codes for COX-2 protein in a mammalian cell, and a pharmaceutically acceptable carrier comprising a pharmaceutically acceptable histidine-lysine polymer. The invention also provides additional methods for using this composition.
The present invention relates to compositions and methods for siRNA therapeutics for prevention and treatment of Middle East Respiratory Syndrome Corona Virus (MERS-CoV) infections. The compositions include a pharmaceutical composition comprising siRNA cocktails that target viral genes and pharmaceutically acceptable polymeric nanoparticle carriers and liposomal nanoparticle carriers.
Disclosed herein are small interfering RNA (siRNA) molecules and pharmaceutical compositions containing them for the prevention and treatment of Ebola virus disease. The present invention provides siRNA molecules that inhibit Ebola virus gene expression, compositions containing the molecules, and methods of using the molecules and compositions to prevent or treat EVD in a subject, such as a human patient.
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
88.
Compositions and methods for “resistance-proof” SiRNA therapeutics for influenza
The present invention relates to compositions and methods for development of resistance-proof siRNA therapeutics for prevention and treatment of influenza viral infections. The compositions include a pharmaceutical composition comprising siRNA molecules that target conserved regions of an influenza virus gene and a pharmaceutically acceptable polymeric carrier. In one embodiment, the polymeric carrier condenses the molecules to form a nanoparticle.
The present invention relates to compositions and methods for development of resistance-proof siRNA therapeutics for prevention and treatment of influenza viral infections. The compositions include a pharmaceutical composition comprising siRNA molecules that target conserved regions of an influenza virus gene and a pharmaceutically acceptable polymeric carrier. In one embodiment, the polymeric carrier condenses the molecules to form a nanoparticle.
The invention provides siRNA compositions that (1) interfere with viral replication of human papillomavirus (HPV), herpes simplex virus (HSV), and human immunodeficiency virus (HIV) in mucosal tissues, such as genital tissues, and (2) treat fungal infections. The compositions include siRNA molecules that target HPV, complexed with a dendrimer that treats and prevents genital herpes (HSV) and HIV. The compositions also include siRNA molecules that target HPV, complexed with a histidine-lysine (HK) polymer that treats and prevent fungus infection. The combined formulations of siRNA and dendrimer provide treatment of the infections from HPVs, HSVs, and HIVs. The combined formulations of siRNA and HK polymer provide treatment of HPVs and fungus infections.
The present invention provides compositions and methods for using combinations of TGFβ1 and Cox-2 inhibitors and TGFβ1 and Hoxb13 inhibitors for the treatment of various medical conditions, including skin scaring due to trauma wounds and surgery, corneal and retina scaring due to injury and surgery, internal organ scaring due to injury and surgery, heart tissue scaring due to heart attack and surgery, and lung, liver, and kidney fibrosis due to inflammation and injury. One example is to use siRNA inhibitors to silence TGFβ1 and Cox-2 at the same time, resulting in significant less scar formation.
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
92.
Compositions and methods using siRNA molecules and siRNA cocktails for the treatment of breast cancer
The present invention provides small interfering RNA (siRNA) molecules, compositions containing the molecules, and methods of using the molecules and compositions to treat breast cancer. In one aspect, a multi-targeted siRNAi cocktail is disclosed. The siRNA molecules may be encapsulated in nanoparticles to further enhance their anti-cancer activity. The compositions may also be used in combination with other anti-cancer agents, such as bevacizumab.
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
93.
SIRNA COMPOSITIONS AND METHODS FOR TREATMENT OF HPV AND OTHER INFECTIONS
The invention provides siRNA compositions that (1) interfere with viral replication of human papillomavirus (HPV), herpes simplex virus (HSV), and human immunodeficiency virus (HIV) in mucosal tissues, such as genital tissues, and (2) treat fungal infections. The compositions include siRNA molecules that target HPV, complexed with a dendrimer that treats and prevents genital herpes (HSV) and HIV. The compositions also include siRNA molecules that target HPV, complexed with a histidine-lysine (HK) polymer that treats and prevent fungus infection. The combined formulations of siRNA and dendrimer provide treatment of the infections from HPVs, HSVs, and HIVs. The combined formulations of siRNA and HK polymer provide treatment of HPVs and fungus infections.
The present invention provides compositions and methods for using a combination of TGFβi and Cox-2 inhibitors for the treatment of various medical conditions including skin scaring due to trauma wounds and surgery, corneal and retina scaring due to injury and surgery, internal organ scaring due to injury and surgery, heart tissue scaring due to heart attack and surgery, and lung, liver, and kidney fibrosis due to inflammation and injury. One example is to use siRNA inhibitors to silence TGFβi and Cox-2 at the same time, resulting in significant less scar formation.
The present invention provides small interfering RNA (siRNA) molecules, compositions containing the molecules, and methods of using the compositions to treat gliomas.
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 5/00 - Peptides ayant jusqu'à quatre amino-acides dans une séquence entièrement déterminéeLeurs dérivés
96.
RARE EARTH-DOPED UP-CONVERSION NANOPARTICLES FOR THERAPEUTIC AND DIAGNOSTIC APPLICATIONS
This invention provides a composition matter comprising rare earth-doped up-conversion nanoparticles (UCNPs) encapsulated with a silica shell. In one embodiment, a photosensitizer is incorporated into the silica shell. In another embodiment, the composition further comprises a targeting molecule. In still another embodiment, a small interfering RNA (siRNA) molecule is also attached to the silica shell with the targeting molecule. The invention further provides methods for synthesizing such compositions and for using them in therapeutic and diagnostic applications. These applications use infrared or near infrared activation to excite the UCNPs.
G06K 7/10 - Méthodes ou dispositions pour la lecture de supports d'enregistrement par radiation électromagnétique, p. ex. lecture optiqueMéthodes ou dispositions pour la lecture de supports d'enregistrement par radiation corpusculaire
97.
Multi-targeted RNAi therapeutics for scarless wound healing of skin
The present invention provides small interfering RNA (siRNA) molecules, compositions containing them, and methods of using them for improvement of skin scarless wound healing and other skin conditions, such as psoriasis and lupus-caused cutaneous lesions. The invention includes siRNA molecules and compositions containing them that inhibit the expression of one or more genes that promote pathological or undesired processes in wound healing and methods of using them.
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
98.
COMPOSITIONS AND METHODS USING SIRNA MOLECULES AND SIRNA COCKTAILS FOR THE TREATMENT OF BREAST CANCER
The present invention provides small interfering RNA (siRNA) molecules, compositions containing the molecules, and methods of using the molecules and compositions to treat breast cancer. In one aspect, a multi -targeted siRNAi cocktail is disclosed. The siRNA molecules may be encapsulated in nanoparticles to further enhance their anti-cancer activity. The compositions may also be used in combination with other anti-cancer agents, such as bevacizumab.
The present invention provides small interfering RNA (siRNA) molecules, compositions containing the molecules, and methods of using the compositions to treat gliomas.
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
100.
MULTI-TARGETED RNAI THERAPEUTICS FOR SCARLESS WOUND HEALING OF SKIN
The present invention provides small interfering RNA (siRNA) molecules, compositions containing them, and methods of using them for improvement of skin scarless wound healing and other skin conditions, such as psoriasis and lupus-caused cutaneous lesions. The invention includes siRNA molecules and compositions containing them that inhibit the expression of one or more genes that promote pathological or undesired processes in wound healing and methods of using them.
